Issued by
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (http://www.nichd.nih.gov)

Purpose

The NICHD invites all stakeholders to submit nominations for research to be prioritized in the development of the BPCA Program’s 2010-11 Priority List of Needs in Pediatric Therapeutics. The NICHD is gathering nominations for a Therapeutic Area (e.g., pediatric condition, subpopulation or setting of care) that requires further study in children as well as the particular drug, biologic, or medical device that is being recommended for study in that area. Suggested topics not related to a specific drug, biologic or device (e.g., training or research collaborations) will be considered by the NICHD outside of this framework.

This is a time-sensitive Request for Information (RFI). There will be two opportunities for public input in 2010:

The nomination form will be available through May 30, 2010. All responses will be compiled into a database that will be reviewed by a committee of stakeholder representatives. The review will result in a preliminary priority list.

There will be a comment period available in July/August 2010 to receive additional public input on the preliminary priority list.

The purpose of this RFI is to obtain knowledge and gather information regarding the current gaps in pediatric therapeutics. The information from this RFI will be used for prioritization planning purposes only and shall not be construed as a solicitation for applications, abstracts, quotations, or as an obligation on the part of the NICHD to provide any funds on the basis of responses.

Background

The NICHD, part of the National Institutes of Health (NIH), leads a pediatric drug development program to identify and prioritize needs in pediatric therapeutics under the Best Pharmaceuticals for Children Act (BPCA). The BPCA was originally authorized in 2002 and was reauthorized in 2007 as part of the Food and Drug Administration Amendments Act. The legislation has two components:

Encouragement of pediatric drug testing through incentives to private companies to conduct research in drugs used in children.

Authorization of a research program through the Department of Health and Human Services (HHS), with the responsibility for the research program implementation through the NIH, specifically the NICHD. The NICHD is responsible for the development of a priority list of needs in pediatric therapeutics, in consultation with the FDA and experts in pediatrics.

Nominations for the BPCA Priority List of Needs in Pediatric Therapeutics are solicited as a part of fulfilling NIH’s authority and responsibility to establish a program for pediatric drug testing and development as outlined in the BPCA legislation. The BPCA Priority List consists of key therapeutic needs in the medical treatment of children and adolescents; it is organized by Therapeutic Area, which can be a group of conditions, a subgroup of the population, or a setting of care. Each calendar year, a few Therapeutic Areas are selected for discussion and further prioritization.

For your reference, the 2008-2009 BPCA Priority List of Pediatric Needs is located on the BPCA website: http://bpca.nichd.nih.gov/about/process/status.cfm. The therapeutic areas of focus for 2008-2009 included: Adolescent Therapeutics, Safety of Atypical Antipsychotic Medications, and Cold & Cough Medicines in Children.

Information Requested

The NICHD is interested in concrete and relevant ideas and approaches to addressing scientific and regulatory needs in therapeutic areas, drugs, biologics, and devices in an effort to best target clinical (or pre-clinical) studies sponsored through the BPCA Program. Nominations should focus on identifying the scientific opportunity and approaches, technology and expertise useful for a project’s development rather than on a funding instrument or solicitation design.

The BPCA nomination form consists of ten comment areas and was developed to solicit information pertinent to specific evaluation criteria, described below.

Please note that with the exception of individuals' email addresses and phone numbers, all responses will also be placed in a public access file. If you choose to omit contact information, your response will still be given full consideration.

Evaluation Criteria

All nominations will be reviewed and evaluated on six key criteria, as follows:

Relevant to BPCA Mission & Goals
No disqualifying ethical concerns
Evidence: consideration of the level of evidence available and current gaps
Impact: potential effect on children, society, and delivery of care
Population: consideration of the different populations that may benefit from the research
Feasibility: consideration of the resources available to conduct the study

If your nomination meets five of the six criteria, your nomination will not be disqualified.

Prioritization Process

NICHD has established a prioritization process through which all nominations will be considered and evaluated by leading pediatric experts and stakeholder representatives.

Gather Nominations (April/May 2010)
NICHD is soliciting research nominations in March 2010 by interacting with key stakeholder groups and requesting submission of the nomination form from the public and other stakeholders.

Prioritize Therapeutic Areas (June 2010)
NICHD will convene key stakeholders to evaluate and prioritize the Therapeutic Area nominations. From this assessment, NICHD will finalize the top Therapeutic Areas for the next Priority List of Pediatric Needs.

Prioritize Pediatric Needs (July 2010)
NICHD will convene one expert workgroup for each of the top Therapeutic Areas. These workgroups will evaluate and prioritize the Pediatric Need nominations within these identified Therapeutic Areas. From this assessment, NICHD will finalize the BPCA Priority List of Pediatric Needs for the calendar year.

Public Comment (July/August 2010)
The Priority List will be available for public comment.

Responses

Submissions in response to this RFI will be considered by the NICHD, the FDA, and an ad hoc evaluation panel to be convened by the NICHD as part of this prioritization process. Nominations will be accepted through May 30, 2010. This nomination form is the only vehicle by which nominations for therapeutic areas, drugs, biologics, and devices will be collected. The form corresponds to one nomination; a party may complete the form multiple times if interested in submitting more than one nomination.

The preferred format for completion of the nomination form is an online form, available at http://www.surveymonkey.com/s/BPCAPriorityListNomination. As an alternative, the nomination form is also available as a PDF document by request to Cynthia Schuster (at the contact information below). The completed PDF form can be completed and sent via email, fax, or mail per the following contact information:

Emails may be sent to Cynthia Schuster at cynthia.schuster@lewin.com. Please indicate “BPCA Nomination” as the subject line of your email.

Faxed responses should be submitted to (703) 269-5501 with attention to Cynthia Schuster..

Letter responses should be addressed to the attention of Cynthia Schuster at the address below: